AUSTIN, Texas--(BUSINESS WIRE)--IntraBio Inc., a leader in the discovery and development of innovative drugs for rare neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved AQNEURSA™ (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.

德克萨斯州奥斯汀——（商业新闻短讯）——发现和开发罕见神经退行性疾病创新药物的领导者IntraBio Inc.今天宣布，美国食品和药物管理局（FDA）已批准AQNEURSA™（左旋乙酰亮氨酸）用于治疗体重≥15公斤的成人和儿科患者C型尼曼匹克病（NPC）的神经系统表现。

AQNEURSA is the only FDA-approved stand-alone therapy indicated for the treatment of NPC..

AQNEUSA是FDA批准的唯一用于治疗NPC的独立疗法。。

'IntraBio has been dedicated to bringing novel treatments to patients with extremely high unmet medical needs like NPC, and today we celebrate a major milestone in this tremendous effort,” said Mallory Factor, President and Chief Executive Officer of IntraBio. “Patients and families in the NPC community have long awaited an effective, FDA-approved treatment, and we are proud to bring hope to those affected by this devastating disease.

IntraBio总裁兼首席执行官马洛里·法因特（MalloryFactor）表示：“IntraBio一直致力于为NPC等医疗需求未得到满足的患者带来新的治疗方法，今天我们庆祝这一巨大努力的一个重要里程碑。NPC社区的患者和家属期待着FDA批准的有效治疗方法，我们很自豪能为受这种毁灭性疾病影响的患者带来希望。

We remain committed to ensuring that all patients who can benefit from this novel treatment will have the opportunity to do so. Based on our clinical research, we believe that AQNEURSA may hold potential for treating other rare and common neurodegenerative and neurodevelopmental disorders, and we will continue to rapidly develop AQNEURSA for these additional indications.”.

我们仍然致力于确保所有能够从这种新型治疗中受益的患者都有机会这样做。根据我们的临床研究，我们相信AQNEUSA可能具有治疗其他罕见和常见的神经退行性疾病和神经发育障碍的潜力，我们将继续迅速开发AQNEUSA用于这些其他适应症。”。

NPC is a rare, inherited lysosomal disease that occurs in about 1 in 100,000 live births. Patients with NPC typically experience systemic, neurological and psychiatric symptoms that can be debilitating and significantly impact functional abilities. Until now, current treatment approaches have not addressed the debilitating effects of NPC on patients’ daily lives..

NPC是一种罕见的遗传性溶酶体疾病，约每100000例活产中就有1例发生。NPC患者通常会出现全身，神经和精神症状，这些症状可能会使人衰弱并严重影响功能。。。

“The FDA approval of AQNEURSA marks a significant breakthrough for those living with Niemann-Pick disease type C,' commented Laurie Turner, Family Services Manager at the National Niemann-Pick Disease Foundation. 'For too long, our community has been without an approved therapy for the treatment of NPC.

美国国家尼曼匹克病基金会家庭服务经理劳里·特纳（LaurieTurner）评论道：“美国食品和药物管理局（FDA）批准的AQNEURSA标志着C型尼曼匹克病患者取得了重大突破。长期以来，我们的社区一直没有批准用于治疗NPC的疗法。

Today we celebrate this tremendous milestone for individuals and families living with NPC. We are immensely thankful for the dedication to innovative research that has led to this approval, and we are ready to help families embark on this new chapter of treatment.”.

今天，我们为与NPC生活在一起的个人和家庭庆祝这一重大里程碑。我们非常感谢对创新研究的奉献，这导致了这一批准，我们准备帮助家庭开启治疗的新篇章。”。

The FDA approval is based on data from the IB1001-301 multinational, randomized, double-blind, placebo-controlled, pivotal clinical trial (NCT05163288), which evaluated the impact of AQNEURSA on neurological symptoms and functioning in pediatric (aged 4 years and older) and adult patients (n=60) with a confirmed diagnosis of NPC..

FDA的批准基于IB1001-301多国随机双盲安慰剂对照关键临床试验（NCT05163288）的数据，该试验评估了AQNEUSA对确诊为NPC的儿科（4岁及以上）和成年患者（n=60）神经症状和功能的影响。。

The trial met the primary efficacy endpoint and all secondary endpoints across all cohorts receiving AQNEURSA. Results from the study showed AQNEURSA significantly improved neurological signs and symptoms and demonstrated functional benefits important to everyday life that were evident within 12 weeks.

该试验符合接受AQNEUSA的所有队列的主要疗效终点和所有次要终点。研究结果显示，AQNEUSA显着改善了神经系统体征和症状，并显示出对日常生活重要的功能益处，这在12周内很明显。

These findings were published in the February 1, 2024 issue of the New England Journal of Medicine..

这些发现发表在2024年2月1日的《新英格兰医学杂志》上。。

The primary outcome assessed by the FDA was a modified version of the Scale for the Assessment and Rating of Ataxia (SARA), referred to as the functional SARA (fSARA). SARA is a clinical assessment tool that assesses gait, stability, speech, and upper and lower limb coordination across eight individual domains.

FDA评估的主要结果是共济失调评估和评定量表（SARA）的修改版本，称为功能性SARA（fSARA）。SARA是一种临床评估工具，可评估八个单独领域的步态，稳定性，言语以及上下肢协调性。

fSARA consists only of gait, sitting, stance, and speech disturbance domains of the original SARA with modifications to the scoring responses. The results in patients who received AQNEURSA compared to placebo showed a greater improvement in fSARA score with a mean treatment difference of -0.4 (95% CI: -0.7, -0.2) with a two-sided p-value of <0.001.

fSARA仅由原始SARA的步态，坐姿，姿势和言语障碍域组成，并对评分反应进行了修改。接受AQNEUSA治疗的患者与安慰剂组相比，fSARA评分有较大改善，平均治疗差异为-0.4（95%CI:-0.7，-0.2），双侧p值小于0.001。

Results on the fSARA were supported by consistent results demonstrated on the original SARA..

fSARA上的结果得到了原始SARA上显示的一致结果的支持。。

AQNEURSA was well tolerated in the trial with the most common adverse reactions (incidence ≥5% and greater than placebo in Period I of the trial) being abdominal pain, dysphagia, upper respiratory tract infections, and vomiting.

AQNEUSA在试验中耐受性良好，最常见的不良反应（发生率≥5%，在试验的第一阶段大于安慰剂）是腹痛，吞咽困难，上呼吸道感染和呕吐。

Indication

指示

AQNEURSA™ (levacetylleucine) is indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg.

AQNEURSA™（左旋乙酰亮氨酸）适用于治疗体重≥15公斤的成人和儿科患者的C型尼曼匹克病（NPC）的神经系统表现。

About IntraBio

关于IntraBio

IntraBio Inc., a US biopharmaceutical company, is focused on the development of novel drugs addressing rare and common neurological diseases. IntraBio's platform technologies result from decades of research and collaboration with universities and institutions worldwide. Its clinical programs are based upon the expertise in lysosomal function and intracellular signaling of its scientific founders from the University of Oxford and the University of Munich..

美国生物制药公司IntraBio Inc.专注于开发治疗罕见和常见神经系统疾病的新药。IntraBio的平台技术是与世界各地的大学和机构进行数十年研究和合作的结果。其临床项目基于牛津大学和慕尼黑大学科学创始人在溶酶体功能和细胞内信号传导方面的专业知识。。